A new clinical trial will test three existing drugs—telmisartan, terazosin, and UDCA—against a shared placebo to see if any can slow Parkinson’s disease progression. Parkinson’s affects roughly 172,000 people in the UK by 2030, yet no treatment exists that modifies the disease itself. Current therapies only manage symptoms and lose effectiveness over time. Previous trials for disease-modifying drugs have been painfully slow, often taking over a decade to complete phase 3 testing, and have failed to recruit a representative patient population. This trial uses a multi-arm, multi-stage design that tests several drugs in parallel, with the option to drop ineffective arms early. It will recruit 1,600 participants from 40 UK sites, with remote or in-person visits over 36 months. If any of the three drugs slows progression by at least 30%, it would meaningfully extend independence for people with Parkinson’s and reduce long-term care costs. The trial also builds a permanent infrastructure for testing future therapies, potentially accelerating the entire field. Results will be shared through patient updates, medical journals, and the UK’s Innovative Licensing Access Pathway to speed regulatory approval.
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This study is funded by the Efficacy and Mechanism Evaluation (EME) Programme, an MRC and NIHR partnership, Cure Parkinson’s, The Michael J Fox Foundation, Parkinson’s UK, The John Black Charitable Foundation, The Gatsby Charitable Foundation and Van Andel Institute. Research question: Do telmisartan, terazosin or UDCA slow the rate of progression of Parkinson’s disease symptoms compared with a shared placebo arm in a multi-arm, multi-stage trial design? Background: Parkinson’s disease (PD) is expected to affect approximately 172,000 people in the UK by 2030. Currently, only symptomatic therapies exist, which become less effective over time. Identification of a disease modifying therapy (DMT) for PD is an urgent unmet need. Previous clinical trials assessing potential DMTs have been hugely inefficient, resulting in timeframes of >10 years for a single therapy to complete phase 3 assessment. Additionally, trial populations are not representative of the UK PD population, limiting the generalisability of findings. Increased efficiency of clinical trial conduct has been successfully demonstrated in multi-arm, multi-stage (MAMS) trials, which assess multiple therapies in parallel, with seamless continuation to phase 3 for therapies with signal of efficacy at early stage analyses. Involving >90 key stakeholders from across the UK, including people with PD (PwP) and care partners, the Edmond J Safra Accelerating Clinical Trials in Parkinson’s Disease (EJS ACT-PD) initiative has produced an inclusive protocol for a Phase 3 multi-centre, MAMS, randomised, double-blind, placebo-controlled trial to assess the clinical and cost effectiveness of potential DMTs in a population representative of PwP in the UK, addressing trial design inefficiencies. Aims: -To assess the clinical and cost-effectiveness of at least 3 potential DMTs compared with a shared placebo on the rate of progression of PD. -To establish sustainable infrastructure for the long-term evaluation of clinical and cost effectiveness of further potential DMTs in PD. -To recruit a representative study population. Methods: We will recruit 1600 participants from 40 UK sites over 3-4 years. Participants will be randomised 1:1:1:1 to one of 3 active treatment arms or placebo. The primary outcome is rate of change in MDS-UPDRS parts I and II combined. Early stage analyses will be undertaken at approximately 2 and 3 years following trial opening, with potential for discontinuation of therapy arms which do not fulfil continuation criteria. Formal efficacy analysis will be undertaken when 266 participants from each trial arm have completed 36 months of treatment. Fifteen core-funded research staff will support delivery at key sites in different UK regions to maximise inclusivity and participant retention. Study visits will be at 3 months, 6 months and then 6-monthly for a total treatment duration of 36 months and be deliverable remotely or in-person. The trial delivery infrastructure that we create will support the addition of future therapy trial arms. Timelines: Q1 2025 - Q4 2028: Participant recruitment Q4 2027: Stage 1 analysis Q4 2028: Stage 2 analysis Q4 2029: Initial efficacy analysis Q4 2030 - Q1 2031: Primary efficacy analysis Q1 - Q2 2031: Results dissemination Anticipated impact and dissemination: The identification of a DMT for Parkinson’s disease would be a major breakthrough with huge clinical impact. Slowing disease progression by at least 30% would result in a meaningful difference in the lives of PwP, increasing independence and decreasing long term care needs and treatment costs. Results will be disseminated to participants in regular study updates, posted online and published in medical journals. The Innovative Licensing Access Pathway (ILAP) will facilitate translation to patient access and care impact.
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